title: Subependymal giant cell astrocytomas in patients with tuberous sclerosis complex: considerations for surgical or pharmacotherapeutic intervention.
abstract: Tuberous sclerosis complex is a genetic disorder caused by mutations in either the TSC1 or TSC2 gene that can result in the growth of hamartomas in multiple organ systems. Subependymal giant cell astrocytomas are slow-growing brain tumors associated primarily with tuberous sclerosis complex. They are usually located in the ventricles, often near the foramen of Monro, where they can cause an obstruction if they grow too large, leading to increased intracranial pressure. Surgery to remove a tumor has been the mainstay of treatment but can be associated with postoperative morbidity and mortality. Not all tumors and/or patients are suitable for surgery. The recent development of mammalian target of rapamycin inhibitors that target the pathway affected by TSC1/TSC2 mutations offers a novel pharmacotherapeutic option for these patients. We review the timing and use of surgery versus pharmacotherapy for the treatment of subependymal giant cell astrocytoma in patients with tuberous sclerosis complex.
authors.affiliation: Department of Pediatric Neurology, Neuroscience Institute and Tuberous Sclerosis Clinic, Le Bonheur Children's Hospital, University of Tennessee Health Science Center, Memphis, TN, USA jwheless@uthsc.edu.
authors.forename: James W
authors.initials: JW
authors.lastname: Wheless
authors.affiliation: Department of Neurosurgery, Neuroscience Institute and Tuberous Sclerosis Clinic, Le Bonheur Children's Hospital, University of Tennessee Health Science Center, Memphis, TN, USA Semmes-Murphey Neurologic & Spine Institute, Memphis, TN, USA St. Jude Children's Research Hospital, Memphis, TN, USA.
authors.forename: Paul
authors.initials: P
authors.lastname: Klimo
mesh_terms: D001254:Astrocytoma / Q000150:complications / Q000188:drug therapy* / Q000601:surgery*; D001932:Brain Neoplasms / Q000150:complications / Q000188:drug therapy* / Q000601:surgery*; D006801:Humans; D007223:Infant; D058570:TOR Serine-Threonine Kinases / Q000037:antagonists & inhibitors; D014402:Tuberous Sclerosis / Q000150:complications* / Q000235:genetics
chemical_list: D058570:TOR Serine-Threonine Kinases
keywords: subependymal giant cell astrocytoma; treatment options; tuberous sclerosis complex